Intellia Therapeutics Announces Three Oral Presentations on In Vivo and Engineered Cell Therapy Data at the 22nd Annual Meeti...
April 15 2019 - 3:30PM
Intellia Therapeutics, Inc. (NASDAQ:NTLA), announced three oral
presentations at the 22nd Annual Meeting of the American Society of
Gene and Cell Therapy (ASGCT), taking place April 29-May 2, 2019,
in Washington, D.C.
Intellia’s data includes important updates from the company’s
programs and platform development activities:
“CRISPR/Cas9-Mediated Targeted Insertion of Human F9
Achieves Therapeutic Circulating Protein Levels in Mice and
Non-Human Primates”
Intellia will present data showing that its targeted gene
insertion platform achieved therapeutic levels of Factor IX protein
in non-human primates (NHP). The company employs a proprietary
hybrid delivery system, comprised of both lipid nanoparticles
(LNPs) and adeno-associated virus (AAV), to insert the desired gene
sequence. Factor 9 (F9) is a gene that encodes Factor IX
(FIX), a blood-clotting protein that is missing or defective in
hemophilia B patients.
The data showing therapeutic levels of FIX achieved in NHPs is
from an ongoing research collaboration
between Intellia and Regeneron Pharmaceuticals,
Inc.
|
Presenter: Hon-Ren Huang, Ph.D., associate
director, Vector Biology, IntelliaAbstract number:
11Session: Advances in Genome Editing and
Hemophilia Gene TherapiesPresentation date/time:
Mon., April 29, 2019, 9-9:15 a.m. ETLocation:
Heights Courtyard 2 |
“Exploiting Clonal Tracking of WT1-Specific T Cells to
Generate a Library of Tumor-Specific T Cell Receptors (TCR) for TCR
Gene Editing of Acute Leukemia”
This presentation will focus on Intellia’s ongoing research
collaboration with IRCCS Ospedale San Raffaele in Italy to develop
CRISPR-edited T cell therapies to address intractable cancers, such
as acute myeloid leukemia (AML). Researchers generated and tested a
library of TCRs with different epitope specificities and human
leukocyte antigen (HLA) restrictions.
|
Presenter: Eliana Ruggiero, Ph.D., Experimental
Hematology Unit, Division of Immunology, Transplantation and
Infectious Diseases, IRCCS Ospedale San Raffaele,
ItalyAbstract number: 123Session:
Cancer Adoptive ImmunotherapyPresentation
date/time: Mon., April 29, 2019, 5-5:15 p.m.
ETLocation: Georgetown |
“CRISPR/Cas9-Mediated Gene Knockout to Address Primary
Hyperoxaluria”
Intellia will provide information demonstrating successful
knockout of two targets of interest, lactate dehydrogenase A (LDHA)
and hydroxyacid oxidase 1 (HAO1), to address primary hyperoxaluria
type 1 (PH1) in a PH1 mouse model. The data shows the continued
progression of the company’s modular platform capability using
CRISPR to knock out liver gene targets. The data being presented
includes results from an ongoing collaboration with researchers at
the University of Alabama at Birmingham.
|
Presenter: Anette Hübner, Ph.D., associate
director, Liver Biology, IntelliaAbstract number:
1000Session: Use of New Technologies for Hepatic
TherapyPresentation date/time: Thur., May 2, 2019,
12-12:15 p.m. ETLocation: Heights Courtyard 3 |
“Delivering on the Therapeutic Promise of
CRISPR/Cas9”
Intellia also will participate in the ASGCT Gene Editing
Workshop, which will provide an overview of current gene editing
technologies and approaches, as well as emerging uses and
applications.
|
Presenter: Sean Burns, M.D., senior director,
Hematology and New Therapeutic AreasSession:
Corporate Review IISession date/time: Sun., April
28, 2019, 5-6 p.m. ETLocation: Lincoln |
Abstracts are available on the ASGCT website.
About Intellia Therapeutics
Intellia Therapeutics is a leading genome editing company
focused on developing curative therapeutics using the CRISPR/Cas9
system. Intellia believes the CRISPR/Cas9 technology has the
potential to transform medicine by permanently editing
disease-associated genes in the human body with a single treatment
course, and through improved cell therapies that can treat cancer
and immunological diseases, or can replace patients’ diseased
cells. The combination of deep scientific, technical and clinical
development experience, along with its leading intellectual
property portfolio, puts Intellia in a unique position to unlock
broad therapeutic applications of the CRISPR/Cas9 technology and
create a new class of therapeutic products. Learn more
about Intellia Therapeutics and CRISPR/Cas9
at intelliatx.com and follow us on Twitter
@intelliatweets.
Forward-Looking Statements
This press release contains "forward-looking statements" of
Intellia within the meaning of the Private Securities Litigation
Reform Act of 1995. These forward-looking statements include, but
are not limited to, express or implied statements regarding our
ability to advance and expand the CRISPR/Cas9 technology to develop
into human therapeutic products, as well as our CRISPR/Cas9
intellectual property portfolio; our ability to achieve stable or
effective genome editing with a single treatment dose; our ability
to perform genomic editing, such as knock-out and insertion, to
treat disease by modulating, replacing or correcting genetic
function; the potential timing and advancement of our preclinical
studies, including continuing non-human primate studies, and
clinical trials; our ability to replicate results achieved in our
preclinical studies in any future studies, including human clinical
trials; the potential development of ex vivo cell therapeutics of
all types using CRISPR/Cas9 technology; our ability to commence
IND-enabling studies of a lead TTR development candidate in 2019
and subsequently submitting an Investigational New Drug
application; our intent to present additional data for our liver
programs, organs beyond the liver, additional insertion/repair
data, and preclinical data in support of our in vivo programs,
including TTR and PH1, as well as our ex vivo programs on
immuno-oncology, including WT1, during 2019; our ability to
nominate a development candidate for an ex vivo program, as well as
a second in vivo indication, in 2019; the intellectual property
position and strategy of Intellia, Intellia’s licensors and other
third parties from which Intellia derived rights; actions by
government agencies; the impact of our collaborations on our
development programs; the potential timing of regulatory filings
regarding our development programs; the potential commercialization
opportunities, including value and market, for product candidates;
our expectations regarding our uses of capital, expenses, future
accumulated deficit and other 2019 financial results; and our
ability to fund operations into the first half of 2021. Any
forward-looking statements in this press release are based on
management’s current expectations and beliefs of future events, and
are subject to a number of risks and uncertainties that could cause
actual results to differ materially and adversely from those set
forth in or implied by such forward-looking statements. These risks
and uncertainties include, but are not limited to: risks related to
Intellia’s ability to protect and maintain our intellectual
property position; risks related to the ability of our licensors to
protect and maintain their intellectual property position;
uncertainties related to the initiation and conduct of studies and
other development requirements for our product candidates; the risk
that any one or more of Intellia’s product candidates will not be
successfully developed and commercialized; the risk that the
results of preclinical studies will be predictive of future results
in connection with future studies; and the risk that Intellia’s
collaborations with Novartis, Regeneron, IRCCS Ospedale San
Raffaele or other collaborations will not continue or will not be
successful. For a discussion of these and other risks and
uncertainties, and other important factors, any of which could
cause Intellia’s actual results to differ from those contained in
the forward-looking statements, see the section entitled “Risk
Factors” in Intellia’s most recent annual report on Form 10-K filed
with the Securities and Exchange Commission, as well as discussions
of potential risks, uncertainties, and other important factors in
Intellia’s other filings with the Securities and Exchange
Commission. All information in this press release is as of the date
of the release, and Intellia Therapeutics undertakes no duty to
update this information unless required by law.
Intellia Contacts:
Media:Jennifer Mound SmoterSenior Vice
PresidentExternal Affairs & Communications+1
857-706-1071jenn.smoter@intelliatx.com
Lynnea OlivarezAssociate DirectorExternal Affairs &
Communications+1
956-330-1917lynnea.olivarez@intelliatx.com
Investors:Lina LiSenior ManagerInvestor
Relations+1 857-706-1612lina.li@intelliatx.com
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